Long-Term Lineage Commitment in Hematopoietic Stem Cell Gene Therapy

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Investigators analyzed hematopoietic reconstitution in 53 patients treated with lentiviral-HSC-gene therapy for diverse conditions such as metachromatic leukodystrophy2,3, Wiskott-Aldrich syndrome4,5, and β-thalassemia6 over a follow-up period of up to eight years, using vector integration sites as markers of clonal identity.
[Nature]
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